Clinical trials: Genetic Mutations
Lung Cancer, Non-small Cell Lung Cancer
Phase II Trial to Correlate Radiographic Response Induced By Gefitinib With Mutations in the Protein-Tyrosine Kinase Domain of the EGF Receptor Gene
The purpose of this research study is to: see if gefitinib pills can shrink Stage 1 or 2 non-small cell lung cancers before surgery see if your non-small cell lung cancer has a mutation in a certain part of the EGFR gene see if…
- Conditions
- Lung Cancer, Non-small Cell Lung Cancer, Bronchioloalveolar Cancer
- Trial name
- NCT00588445
- Testing
- Gefitinib
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Ovarian Cancer
BMN 673 (Talazoparib), an Oral PARP Inhibitor, in People With Deleterious BRCA1/2 Mutation-Associated Ovarian Cancer Who Have Had Prior PARP Inhibitor Treatment
Background: The new drug BMN 673 (talazoparib) has been shown to fight tumor cells in animals and some people. It is a poly (ADP-ribose) polymerase (PARP) inhibitor.
- Trial name
- NCT02326844
- Testing
- BMN 673 (talazoparib)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Cancer Institute (NCI)
Metastatic Non Small Cell Lung Cancer
Dacomitinib for Treatment of Patients in India With Metastatic Non Small Cell Lung Cancer With EGFR Activating Mutations
This is a Phase 4, open label, single arm, multi-center, prospective clinical trial of dacomitinib that will be performed in India.
- Trial name
- NCT04511533
- Testing
- Dacomitinib
- Where
- 15 sites in India
- Volunteers
- 101 people
- Sponsor
- Pfizer
Cystic Fibrosis
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del…
- Trial name
- NCT04353817
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 121 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma
- Trial name
- NCT03220035
- Testing
- Laboratory Biomarker Analysis, Vemurafenib
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- National Cancer Institute (NCI)
Carcinoma, Non-Small-Cell Lung
Afatinib in Patients With Non Small Cell Lung Cancer (NSCLC) With Epidermal Growth Factor Receptor (EGFR) Mutations
The main aim of this study is to evaluate the safety and tolerability of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR) mutation(s)…
- Trial name
- NCT02044380
- Testing
- Afatinib
- Where
- 5 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Boehringer Ingelheim
Cystic Fibrosis
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
- Trial name
- KONTINUE (NCT01707290)
- Testing
- Ivacaftor
- Where
- 38 sites in United States, Belgium and 2 more
- Volunteers
- 125 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Carcinoma, Non-Small-Cell Lung
Study of Capmatinib and Spartalizumab/Placebo in Advanced NSCLC Patients With MET Exon 14 Skipping Mutations
A double-blind, placebo controlled, randomized, phase II study evaluating the efficacy and safety of capmatinib (INC280) and spartalizumab (PDR001) combination therapy versus capmatinib and placebo as first line treatment for…
- Trial name
- NCT04323436
- Testing
- Spartalizumab, Capmatinib
- Where
- 15 sites in United States, Belgium and 7 more
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Breast Cancer
A Phase I, Open-Label, 2 Part Multicentre Study to Assess the Safety and Efficacy of Olaparib in Combination With Carboplatin in Patients With Advanced HER-2 Negative Breast Cancer
This is an open-label study to assess the safety, tolerability and efficacy of olaparib in combination with carboplatin. There are two parts in this study: Part A, a dose escalation in patients with advanced Human Epidermal…
- Trial name
- NCT02561832
- Testing
- Olaparib, Carboplatin, Anthracycline
- Where
- 7 sites in United States, Spain
- Volunteers
- 15 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following…
- Trial name
- KONNECTION (NCT01614470)
- Testing
- Ivacaftor
- Where
- 12 sites in United States, Belgium and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Malignant Melanoma
Comparison of In-House Methods and Cobas BRAF V600 Mutation Assay in Melanoma Tumor Samples
This non-interventional study will compare the Cobas BRAF V600 mutation assay with in-house methods used in molecular laboratories for the assessment of the BRAF mutation status in melanoma tumor samples.
- Trial name
- NCT01744860
- Where
- 12 sites in France
- Volunteers
- 420 people
- Sponsor
- Hoffmann-La Roche
Relapsed Ovarian Cancer, BRCA Mutation, Platinum Sensitivity
Olaparib Treatment in Relapsed Germline Breast Cancer Susceptibility Gene (BRCA) Mutated Ovarian Cancer Patients Who Have Progressed at Least 6 Months After Last Platinum Treatment and Have Received at Least 2 Prior Platinum Treatments
Comparison of olaparib vs. physician's choice of single agent standard of care non-platinum based chemotherapy in patients with germline Breast Cancer susceptibility gene (gBRCA) mutated ovarian cancer who have progressed at…
- Trial name
- SOLO3 (NCT02282020)
- Testing
- OLAPARIB, Single agent chemotherapy
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 266 people
- Sponsor
- AstraZeneca
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non-Small Cell Lung Cancer
A Study of Erlotinib in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer
This open-label, multi-center study will evaluate the progression-free survival and safety of erlotinib in participants with locally advanced or metastatic non-small cell lung cancer with activating mutations in the tyrosine…
- Trial name
- ESSENCE (NCT01667562)
- Testing
- Erlotinib
- Where
- 4 sites in Serbia
- Volunteers
- 375 people
- Sponsor
- Hoffmann-La Roche
Non-Small Cell Lung Cancer
A Study of Tarceva (Erlotinib) in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer Who Present EGFR Mutations
This single arm, open-label study will assess the efficacy and safety of Tarceva (erlotinib) in patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) mutations.
- Trial name
- NCT01287754
- Testing
- erlotinib [Tarceva]
- Where
- 5 sites in Finland
- Volunteers
- 24 people
- Sponsor
- Hoffmann-La Roche
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Solid Tumor
A Study of BDTX-189, an Orally Available Allosteric ErbB Inhibitor, in Patients With Advanced Solid Tumors.
This was a clinical study with an orally administered drug, BDTX-189 in participants with advanced solid tumors that had select mutations or alterations in human epidermal growth factor receptor 2 (HER2/ErbB2) genes or epidermal…
- Trial name
- MasterKey-01 (NCT04209465)
- Testing
- BDTX-189
- Where
- 38 sites in United States, Denmark and 2 more
- Volunteers
- 91 people
- Sponsor
- Black Diamond Therapeutics, Inc.
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics